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시장보고서
상품코드
2082961
골수 이형성 증후군(MDS) : 시장 인사이트, 역학 및 시장 예측(2036년)Myelodysplastic Syndrome - Market Insight, Epidemiology, and Market Forecast - 2036 |
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DelveInsight
본 MDS 시장 보고서에서는 표준 치료, 임상 실무, 진화하는 치료 알고리즘 등 현재 시장 상황에 대해 종합적인 분석을 제시하고 있습니다. 또한, MDS 환자의 부담 추이, 수익 및 시장 점유율 추이, 피크 시기의 환자 점유율 및 치료 도입 현황에 대한 분석을 평가함과 동시에, 세계 각 지역 시장 규모에 대한 상세한 평가 및 성장률 예측(과거 데이터 및 2022년-2036년 예측)을 제공합니다. 본 보고서에서는 MDS 분야의 주요 미충족 요구 사항을 부각시키고, 경쟁 구도 및 임상 현황을 분석하여 고부가가치의 성장 기회를 도출함으로써, 향후 시장 성장 가능성에 대한 명확한 전망을 제시하고 있습니다.
고령화의 진행과 질병 부담 증가
MDS는 주로 고령자에게 발병하며, 65세 이상 인구에서 발병률이 현저히 증가하고 있습니다. 주요 시장의 고령 인구 증가에 따라 MDS의 진단 및 치료 건수가 증가할 것으로 예상되며, 이에 따라 시장 기회가 확대될 것으로 전망됩니다.
병용 요법의 활용 확대
저메틸화제, B세포 림프종-2(Bcl-2) 억제제, 면역조절요법 및 신규 표적 치료제를 이용한 병용요법이 점점 더 높이 평가받고 있습니다. 이러한 전략을 통해 얻은 긍정적인 임상 결과가 치료법 도입을 촉진하고 시장 성장을 이끌 것으로 예측됩니다.
임상시험 활동의 활성화와 파이프라인의 혁신
세포 치료, 면역 치료, 표적 치료제를 특징으로 하는 탄탄한 파이프라인은 MDS에 대한 업계의 높은 관심을 반영하고 있습니다. 새로운 치료법에 대한 지속적인 임상 개발과 승인 가능성으로 인해 치료 선택지가 다양해지고, 시장 확대가 촉진될 것으로 예측됩니다.
골수 이형성 증후군(MDS)의 개요와 진단
MDS는 조혈 기능 부전을 특징으로 하며, 그 결과 정상적인 혈액 세포의 생성이 불충분해지는 드물고 이질성이 높은 골수 질환군입니다. 이 질환은 조혈모세포의 유전적 및 후성유전적 이상에 기인하며, 지속적인 혈구 감소, 골수이형성 및 급성 골수성 백혈병(AML)으로 진행될 위험 증가를 초래합니다. MDS는 주로 고령자에게 발병하며, 만성 빈혈, 재발성 감염, 출혈성 합병증, 삶의 질 저하, 그리고 지속적인 의료 관리가 필요하다는 점으로 인해 큰 임상적 부담을 초래합니다. MDS의 생물학적 및 임상적 이질성으로 인해 위험도에 따른 치료 접근법이 개발되어 왔으나, 여전히 새로운 질환 수정 요법의 필요성이 대두되고 있습니다.
MDS의 진단은 임상 평가, 검사 소견, 골수 검사, 그리고 세포유전학적 또는 분자생물학적 분석을 종합하여 이루어집니다. 환자는 일반적으로 전혈구계수(CBC)를 통해 확인되는 원인을 알 수 없는 지속적인 혈구 감소 증상을 보입니다. 골수 천자 및 생검은 이형성, 블라스트 비율, 세포 밀도를 평가하는 데 필수적이며, 한편 세포유전학적 검사 및 차세대 염기서열 분석(NGS)은 진단, 위험도 분류 및 치료법 선택을 뒷받침하는 염색체 이상과 유전자 변이를 확인하는 데 도움이 됩니다. 예후는 일반적으로 IPSS-R이나 IPSS-M과 같은 점수 산정 시스템을 통해 평가됩니다.
골수 이형성 증후군(MDS)의 현재 치료 현황
MDS의 치료 전략은 질환의 위험도, 증상의 중증도, 연령 및 환자의 전신 상태를 바탕으로 결정됩니다. 저위험군 환자의 경우, 빈혈에 대해 적혈구 수혈, 적혈구 조혈 자극 인자(ESA), 그리고 루스파텔셉트를 포함한 지지 요법이 시행되는 경우가 많습니다. 고위험군 환자에게는 일반적으로 아자시티딘이나 데시타빈과 같은 저메틸화제를 이용한 질환 수정 요법이 시행됩니다. 또한, 적격한 환자의 경우, 유일한 근치적 치료법이 될 수 있는 동종 조혈모세포 이식이 시행되는 경우가 있습니다. Bcl-2 억제제, 면역 표적 치료제, 새로운 세포 치료법 등 새로운 치료법들이 MDS 치료의 현 상황을 한층 더 변화시킬 것으로 기대되고 있습니다.
MDS의 역학 분석 및 예측에 관한 주요 조사 결과
MDS의 치료 환경은 기존의 대증 요법이나 저메틸화제(HMA)에서 특정 분자 이상이나 질환의 생물학적 특성에 대응하도록 설계된 표적 치료, 적혈구 성숙 촉진제, 그리고 병용 요법으로 큰 변화를 겪고 있습니다. 현재 이 시장은 루스파텔셉트-aamt, 이메텔스타트, 이보시데닙 등 승인된 치료제들에 의해 지탱되고 있으며, 이들 각각은 서로 다른 환자층을 차지하고 있습니다. 루스파텔셉트는 저위험 수혈 의존성 빈혈에 대한 1차 치료제로서의 입지를 확고히 하고 있는 반면, 이메텔스타트는 ESA 내성 환자에서 지속적인 수혈 불필요 상태를 실현함으로써 경쟁력을 높이고 있습니다. 이보시데닙은 IDH1 변이를 가진 환자로 제한되지만, MDS에서 바이오마커 기반 치료 접근법의 역할이 확대되고 있음을 보여줍니다. 이메텔스타트는 2026년 1분기에 5,180만 달러의 순매출을 기록했으며, 이는 2025년 4분기 대비 8% 증가한 수치로, 출시 초기 단계부터 견조한 시장 침투세를 보이고 있습니다. 한편, 루스파텔셉트는 적응증 범위가 넓고 시장에 조기에 진출한 덕분에, 여전히 시장에서 매우 큰 입지를 유지하고 있습니다.
신흥 치료법 중에서도 베네토클락스는 HR-MDS 분야에서 가장 영향력 있는 파이프라인 자산 중 하나가 될 것으로 기대되고 있습니다. 이 약물은 이미 급성 골수성 백혈병(AML) 분야에서 확고한 임상적 입지를 다졌으며, 현재 진행 중인 골수 이형성 증후군(MDS) 임상시험에서는 아자시티딘과의 병용 요법을 통해 유망한 반응률이 나타나고 있습니다. 진행 중인 임상시험에서 생존 이점이 입증되고, 안전성이 허용 범위 내에 있음이 확인된다면, 베크마릴리맙은 재발성·난치성 MDS 환자에서 보여준 유망한 효능으로 인해 주목을 받고 있습니다. 초기 임상 데이터에 따르면, 지속적인 치료 효과와 양호한 내약성이 확인되었으며, 이는 치료 선택지가 제한적인 환자 집단에서 이 약물의 잠재적 역할을 뒷받침하고 있습니다. 또 다른 주목할 만한 신흥 자산은 리사프트크라크스로, 병용 요법에서 유망한 효능의 징후가 보고되어 차세대 BCL-2 억제제로 자리매김하고 있습니다. 이 약물의 향후 경쟁력은 안전성, 투여의 유연성, 혹은 효능의 지속성 측면에서 베네토클락스에 비해 우위를 보여줄 수 있느냐에 달려 있습니다.
MDS 분야의 혁신은 더 이상 단일 치료 기전에 의해 주도되는 것이 아니라, 적아구 성숙 촉진제, 텔로머라제 억제제, 표적 분자 치료, 세포 사멸 유도제, 면역 조절제 및 세포 치료법의 융합을 통해 추진되고 있습니다. 이러한 다양화로 인해 치료 알고리즘이 재구축되고, 분자적 및 임상적 특성에 기반한 환자 계층화가 진행되며, 예측 기간 동안 지속적인 시장 성장이 촉진될 것으로 기대됩니다.
DelveInsight's 'Myelodysplastic Syndrome (MDS) - Market Insights, Epidemiology and Market Forecast - 2036' report delivers an in-depth understanding of the MDS, historical and forecasted epidemiology, as well as the MDS market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.
The MDS market report delivers a comprehensive analysis of the current treatment landscape, including standards of care, clinical practices, and evolving therapeutic algorithms. It evaluates MDS patient burden trends, revenue & market share dynamics, peak patient share & therapy uptake analysis, and provides an in-depth market size assessment, and growth rate projections (Historical & Forecast 2022-2036) across global regions. The report highlights key unmet medical needs in MDS and maps the competitive and clinical landscape to uncover high-value opportunities, providing a clear outlook on future market growth potential.
Key Factors Driving the Myelodysplastic Syndrome (MDS) Market
Increasing Aging Population and Disease Burden
MDS predominantly affects older adults, with incidence rising significantly in individuals aged 65 years and above. The growing elderly population across major markets is expected to increase the number of diagnosed and treated MDS cases, thereby expanding the market opportunity.
Expanding Use of Combination Therapies
Combination approaches involving hypomethylating agents, B-cell lymphoma-2 (Bcl-2) inhibitor, immune-modulating therapies, and novel targeted agents are being increasingly evaluated. Positive clinical outcomes from these strategies are expected to drive treatment adoption and market growth.
Growing Clinical Trial Activity and Pipeline Innovation
A robust pipeline featuring cell therapies, immunotherapies, and targeted agents reflects strong industry interest in MDS. Continued clinical development and potential approvals of novel therapies are expected to diversify treatment options and stimulate market expansion.
Myelodysplastic Syndrome (MDS) Overview and Diagnosis
MDS is a group of rare, heterogeneous bone marrow disorders characterized by ineffective hematopoiesis, leading to insufficient production of healthy blood cells. The disease arises from genetic and epigenetic abnormalities in hematopoietic stem cells, resulting in persistent cytopenias, bone marrow dysplasia, and an increased risk of progression to acute myeloid leukemia (AML). MDS primarily affects older adults and presents a substantial clinical burden due to chronic anemia, recurrent infections, bleeding complications, reduced quality of life, and the need for ongoing medical management. The biological and clinical heterogeneity of MDS has led to the development of risk-adapted treatment approaches and continues to drive the need for novel disease-modifying therapies.
The diagnosis of MDS is based on a combination of clinical evaluation, laboratory testing, bone marrow examination, and cytogenetic or molecular analyses. Patients typically present with persistent unexplained cytopenias identified through complete blood counts (CBCs). Bone marrow aspiration and biopsy are essential for assessing dysplasia, blast percentage, and cellularity, while cytogenetic testing and next-generation sequencing (NGS) help identify chromosomal abnormalities and gene mutations that support diagnosis, risk stratification, and treatment selection. Prognosis is commonly assessed using scoring systems such as the IPSS-R and IPSS-M.
Current Myelodysplastic Syndrome (MDS) Treatment Landscape
Treatment strategies for MDS are guided by disease risk, symptom burden, age, and patient fitness. Lower-risk patients are often managed with supportive care, including red blood cell transfusions, erythropoiesis-stimulating agents (ESAs), and luspatercept for anemia. Higher-risk patients generally receive disease-modifying therapies such as hypomethylating agents, including azacitidine and decitabine, while eligible patients may undergo allogeneic hematopoietic stem cell transplantation, the only potentially curative treatment. Emerging therapies such as Bcl-2 inhibitors, immune-targeted agents, and novel cellular therapies are expected to further transform the MDS treatment landscape.
Myelodysplastic Syndrome (MDS) Unmet Needs
The section "unmet needs of MDS" outlines the critical gaps between the current state of patient care, diagnosis, and the ideal & effective management of the disease. It highlights the obstacles experienced by patients, clinicians, and researchers and identifies potential solutions for future progress.
Key Findings from MDS Epidemiological Analysis and Forecast
Myelodysplastic Syndrome (MDS) Drug Analysis & Competitive Landscape
The MDS drug chapter provides a detailed, market-focused review of the emerging pipeline across Phase III/I clinical trials and preclinical trials. It covers the mechanism of action, clinical trial data, regulatory approvals, patents, collaborations, and strategic partnerships for each therapy, along with their advantages, limitations, and recent developments. This section offers critical insights into the MDS treatment landscape, supporting market assessment, competitive analysis, and growth forecasting for the MDS therapeutics market.
Approved Therapies for Myelodysplastic Syndrome (MDS)
Imetelstat (RYTELO): Geron
Imetelstat is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Imetelstat is the first and only telomerase inhibitor approved by the US FDA and the European Commission. It has US market exclusivity till June 2029 and till March 2035 in EU. It also has orphan drug exclusivity till June 2031 in US and till March 2025 in EU. RYTELO is positioned to lead in second-line Lower-Risk Myelodysplastic Syndrome (LR-MDS).
Ivosidenib (TIBSOVO): Servier
Ivosidenib is a oral, targeted, first-in-class precision medicine developed by Servier that blocks the isocitrate dehydrogenase-1 (IDH1) mutation. It is primarily prescribed for adults with relapsed/refractory (r/r) or newly diagnosed MDS. The FDA approval of this indication is supported by a pivotal Phase I, open-label study in IDH1-mutated R/R MDS patients. The therapy has demonstrated clinical benefit in hematologic cancers and targeted oncology settings driven by IDH1 mutations.
Myelodysplastic Syndrome (MDS) Pipeline Analysis
Venetoclax (VENCLEXTA): AbbVie
Venetoclax is a selective Bcl-2 inhibitor. The US FDA granted a Breakthrough Therapy Designation (BTD) to venetoclax in combination with azacitidine for the treatment of adult patients with previously untreated intermediate-, high- and very high-risk MDS. Venetoclax is a prescription medicine used in chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). Venetoclax is being developed by AbbVie and Roche. It is jointly commercialized by Genentech in the US and by AbbVie outside of the US.
Orca-T: OrcaBio
Orca-T is an investigational allogeneic T-cell immunotherapy under evaluation for the treatment MDS. Orca Bio is evaluating Orca-T across multiple clinical trials in MDS, including Phase III, Phase II, and Phase I/Ib studies assessing different donor settings, conditioning regimens, and transplant approaches. By improving transplant outcomes and lowering transplant-related complications, Orca-T has the potential to expand access to curative HSCT for patients with HR-MDS.
Myelodysplastic Syndrome (MDS) Key Players, Market Leaders and Emerging Companies
Myelodysplastic Syndrome (MDS) Drug Updates
The treatment landscape for MDS is undergoing a significant transformation from traditional supportive care and hypomethylating agents (HMAs) toward targeted therapies, erythroid maturation agents, and combination regimens designed to address specific molecular abnormalities and disease biology. The market is currently anchored by approved therapies such as luspatercept-aamt, imetelstat, and ivosidenib, each occupying distinct patient segments. Luspatercept has established itself as a preferred option for lower-risk transfusion-dependent anemia, while imetelstat has strengthened competition by offering durable transfusion independence in ESA-refractory patients. ivosidenib, although limited to IDH1-mutated disease, exemplifies the growing role of biomarker-driven treatment approaches in MDS. Imetelstat generated net revenue of USD 51.8 million in Q1 2026, representing an 8% increase over Q4 2025, indicating strong early commercial uptake. While luspatercept continues to maintain a substantially larger market presence owing to its broader label and earlier market entry.
Among emerging therapies, venetoclax is expected to be one of the most influential pipeline assets in the HR-MDS setting. The drug has already established a strong clinical foundation in AML, and ongoing MDS studies have shown encouraging response rates when combined with azacitidine. If ongoing trials demonstrate a survival benefit and manageable safety profile. Bexmarilimab has attracted attention due to its encouraging activity in patients with r/r MDS. Early clinical data have demonstrated durable responses and a favorable tolerability profile, supporting its potential role in a patient population with limited therapeutic options. Another notable emerging asset is lisaftoclax, which has reported promising efficacy signals in combination regimens and is being positioned as a next-generation BCL-2 inhibitor. Its future competitiveness will depend on whether it can demonstrate advantages over venetoclax in terms of safety, dosing flexibility, or durability of response.
Drug Class/Insights into Leading Emerging and Marketed Therapies in MDS (2022-2036 Forecast)
The MDS market (2022-2036 forecast) is evolving from a landscape historically dominated by supportive care and hypomethylating agents toward a more targeted and biomarker-driven treatment paradigm. Advances in disease biology have led to the development of therapies addressing ineffective hematopoiesis.
MDS innovation is no longer driven by a single therapeutic mechanism but rather by a convergence of erythroid maturation agents, telomerase inhibitors, targeted molecular therapies, apoptosis-inducing agents, immune modulators, and cellular therapies. This diversification is expected to reshape treatment algorithms, increase patient stratification based on molecular and clinical characteristics, and drive sustained market growth throughout the forecast period.
Myelodysplastic Syndrome (MDS) Drug Uptake
This section focuses on the uptake rate of potential drugs expected to be launched in the market during the forecast period (2026-2036). The analysis covers the MDS drug's uptake, performance at peak, factors affecting performance during prime years of growth, patient uptake by therapy, and anticipated sales generated by each drug.
Among approved therapies, luspatercept is expected to maintain strong uptake in LR-MDS patients with anemia, supported by its established efficacy in reducing transfusion burden and its expanding role in earlier lines of treatment. Imetelstat is anticipated to witness increasing adoption among transfusion-dependent LR-MDS patients following ESA failure, driven by its novel telomerase inhibition mechanism and durable transfusion independence outcomes. Meanwhile, Ivosidenib is expected to experience more selective uptake due to its applicability to the relatively small subset of patients harboring IDH1 mutations, although its precision-medicine approach supports strong value within this niche population.
The emerging MDS pipeline is expected to drive increased competition across both lower-risk and higher-risk disease segments. Venetoclax is expected to achieve strong uptake in HR-MDS, supported by its established clinical use in AML and promising combination data with hypomethylating agents. Lisaftoclax (APG-2575) may emerge as a competitive next-generation Bcl-2 inhibitor if it demonstrates advantages in efficacy or tolerability over existing Bcl-2-based regimens. Bexmarilimab is likely to gain traction in R/R MDS, where unmet need remains high, while ofirnoflast and R289 may see selective adoption if their novel immune and inflammatory pathway-targeting approaches translate into meaningful clinical benefit.
Detailed insights of emerging therapies' drug uptake is included in the report
Market Access and Reimbursement of Approved therapies in Myelodysplastic Syndrome (MDS)
The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.
Reimbursement is a crucial factor that affects the drug's access to the market. Often, the decision to reimburse comes down to the price of the drug relative to the benefit it produces in treated patients. To reduce the healthcare burden of these high-cost therapies, many payment models are being considered by payers and other industry insiders.
NOTE: Further Details are provided in the final report....
Myelodysplastic Syndrome (MDS) Therapies Price Scenario & Trends
Pricing and analogue assessment of MDS therapies highlights evolving price dynamics structures. This section summarizes the cost of approved treatments, closest and most appropriate analogue selection for emerging therapies, and understanding of how pricing influences market access, adherence, and long-term uptake.
Industry Experts and Physician Views for Myelodysplastic Syndrome (MDS)
To keep up with MDS market trends, we take Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on the MDS emerging therapies, evolving treatment landscape, patient adherence to conventional therapies, therapy switching trends, drug adoption and uptake, accessibility challenges, and epidemiology and real-world prescription patterns in MDS, including MD, PhD, Instructor, Postdoctoral Researcher, Professor, Researcher, and others.
DelveInsight's analysts connected with 15+ KOLs to gather insights at the country level. Centers such as Stanford MDS Center, Memorial Sloan Kettering Cancer Center, and University Medical Center Mainz etc. were contacted. Their opinion helps understand and validate current and emerging MDS therapies, highlight unmet medical needs, provide epidemiological context, and support strategic decisions for market access, therapy adoption, and pipeline prioritization in MDS.
Qualitative Analysis: SWOT and Conjoint Analysis
We perform qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and conjoint analysis.
In the SWOT analysis of MDS, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.
Conjoint analysis analyzes emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.
The team of analysts analyzes promising emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. In efficacy, the trial's primary and secondary outcome measures are evaluated, whereas the therapies' safety is evaluated, wherein the acceptability, tolerability, and adverse events are majorly observed. In addition, the scoring is also based on the route of administration, order of entry, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.
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